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Efficacy and Safety of Tazbentetol in ALS Participants

ConditionAmyotrophic Lateral Sclerosis
Trial statusNot yet recruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age18-80
SponsorSpinogenix

About this trial

The objectives of this study are to examine the effects of tazbentetol on clinical measures of ALS, patient reported outcomes (PROs), long-term safety and tolerability.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age 18-80

ALS TRICALS risk score

Stable dose of standard of care treatment

Contraception use by men or women consistent with local regulations

Disqualifiers

Underlying physical or psychological condition prohibiting study completion

Clinically significant cardiac disease

Active or history of malignancy in the past 5 years

Serious infection within 1 month of screening

Trial design

Design model

Parallel

Treatments tested in this trial

  • Tazbentetol

    Drug

    Participants in both Phase 2B and Phase 3 will be randomized to received study drug tazbentetol or placebo tablets. Participants in the open-label extension phase will receive the dose determined from Phase 2.

  • placebo

    Drug

    participants in double blind placebo controlled phase will be randomized to received placebo tablets

Treatment groups

430 Participants
are divided into 2 treatment groups
Group A: tazbentetolExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Phase 2: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score

Questionnaire administered by a clinician that measures participants' ability to function in certain daily activities. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.

Time frame
36 weeks
2

Phase 3: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score

Questionnaire administered by a clinician that measures participants' ability to function in certain daily activities. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.

Time frame
36 weeks
3

OLE: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score

Questionnaire administered by a clinician that measures participants' ability to function in certain daily activities. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.

Time frame
36 weeks

Secondary outcomes

1

Phase 2: Combined Assessment of Function and Survival (CAFS) scores

This assessment ranks clinical outcome based on changes to ALSFRS-R score. A higher score indicates a better clinical outcome.

Time frame
36 weeks
2

Phase 2: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)

Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation

Time frame
36 weeks
3

Phase 2: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)

Questionnaire to assesses cognitive and behavioral changes in people with (ALS) through a 136-point test covering language, verbal fluency, executive function, memory, and visuospatial cognitive domains. A lower score indicates worsening of symptoms.

Time frame
36 weeks
4

Phase 2: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)

The ALSAQ40 score is a measure of QoL for patients with ALS. The ALSAQ40 evaluates domains that include physical mobility, activities of daily living (ADL) and independence, eating and drinking, communication, and emotional reactions. A higher score indicates higher disability.

Time frame
36 weeks

Other outcomes

1

Phase 2: ALSFRS-R total score rate of decline

To further assess the effect of tazbentetol on the progression of ALS

Time frame
36 weeks
2

Phase 2: Proportion of patients alive and survival time

To further assess the effect of tazbentetol on the progression of ALS

Time frame
36 weeks
3

Phase 2 substudy: To compare electroencephalogram (EEG) outcomes of two dose levels of tazbentetol to placebo in participants with ALS

To measure the change from baseline in EEG delta and power bands at resting state at Weeks 8, 24, and 36

Time frame
36 weeks
4

Phase 3: ALSFRS-R total score rate of decline

To further assess the effect of tazbentetol on the progression of ALS

Time frame
36 weeks

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Spinogenix

Lead sponsor

Source ClinicalTrials.gov

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